3 active not recruiting, 3 completed, 3 recruiting, 2 terminated. 4 Phase 2, 1 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 4 | Active not recruiting 1, Completed 1, Not yet recruiting 1, Terminated 1 |
| Phase 1 | 1 | Active not recruiting 1 |
Follow Krabbe Disease: new trials by email Literature Review of Krabbe Disease trials Research Report on Krabbe Disease
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients (Master Protocol) PLX-200 - Lysosomal Storage Disorders, Sandhoff Disease | Phase 2 | Not yet recruiting | Polaryx Therapeutics, Inc. | 24 | 2026-12-01 | 2026-07-31 |
| Long-term Follow-up Study to Evaluate Safety and Efficacy of FBX-101 in Krabbe Patients FBX-101 - Krabbe Disease | Terminated | Forge Biologics, Inc | 2 | 2024-08-06 | 2025-09-05 | |
| Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated Previously With HSCT FBX-101 - Krabbe Disease | Phase 1 / Phase 2 | Active not recruiting | Forge Biologics, Inc | 9 | 2023-02-03 | 2025-11-20 |
| Early Check: Expanded Screening in Newborns Confirmatory Testing - Spinal Muscular Atrophy, Fragile X Syndrome | Active not recruiting | RTI International | 30000 | 2018-10-15 | 2026-07-01 | |
| Biomarker for Krabbe Disease (BioKrabbe) Krabbe Disease | Withdrawn | CENTOGENE GmbH Rostock | 2018-08-20 | 2023-02-13 | ||
| LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies Leukodystrophy, White Matter Disease | Completed | Children's Hospital of Philadelphia | 236 | 2017-01-06 | 2025-11-10 | |
| The Myelin Disorders Biorepository Project Leukodystrophy, White Matter Disease | Recruiting | Children's Hospital of Philadelphia | 12000 | 2016-12-08 | 2025-10-23 | |
| Krabbe Disease Global Patient Registry Krabbe Disease | Recruiting | State University of New York at Buffalo | 60 | 2014-09-01 | 2026-07-17 | |
| Longitudinal Study of Neurodegenerative Disorders Palliative Care, Hematopoetic Stem Cell Transplantation - MLD, Krabbe Disease | Recruiting | University of Pittsburgh | 1500 | 2012-01-11 | 2026-02-09 | |
| Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders Campath-1H, Cyclophosphamide, Busulfan - Mucopolysaccharidosis, Hurler Syndrome | Phase 2 | Completed | Masonic Cancer Center, University of Minnesota | 46 | 2009-12-01 | 2018-02-05 |
| Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children Mucopolysaccharidosis Type I (MPS I), Mucopolysaccharidosis Type II (MPS II) | Completed | University of Chicago | 19 | 2009-01-01 | 2019-10-04 | |
| Diffusion Tensor Imaging (DTI) in Infants With Krabbe Disease Krabbe Disease | Active not recruiting | University of Pittsburgh | 100 | 2008-04-01 | 2026-03-06 | |
| Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism Stem Cell Transplantation, Cyclophosphamide, Campath-1H - Hurler's Syndrome, Maroteaux-Lamy Syndrome | Phase 2 | Terminated | Masonic Cancer Center, University of Minnesota | 18 | 2008-03-01 | 2017-12-28 |
Leukodystrophy, Globoid Cell (13) · Leukodystrophy, Metachromatic (5) · Peroxisomal Disorders (5) · Leukoencephalopathies (4) · Lysosomal Storage Diseases (4) · Mucopolysaccharidoses (4) · Mucopolysaccharidosis I (4) · Mucopolysaccharidosis VI (4) · Sphingolipidoses (4) · Adrenoleukodystrophy (3) · Canavan Disease (3) · Mucopolysaccharidosis II (3) · Mucopolysaccharidosis III (3) · Mucopolysaccharidosis VII (3) · Multiple Sulfatase Deficiency Disease (3)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (13) · Nutritional and Metabolic Diseases (13) · Brain Diseases (12) · Brain Diseases, Metabolic (12) · Brain Diseases, Metabolic, Inborn (12) · Central Nervous System Diseases (12) · Demyelinating Diseases (12) · Genetic Diseases, Inborn (12) · Hereditary Central Nervous System Demyelinating Diseases (12) · Lipid Metabolism Disorders (12) · Lipid Metabolism, Inborn Errors (12) · Lipidoses (12) · Lysosomal Storage Diseases, Nervous System (12) · Metabolic Diseases (12) · Nervous System Diseases (12)
Alemtuzumab (2) · Busulfan (2) · Cyclophosphamide (2) · Stem Cell Transplantation (2) · Bone Marrow Transplantation (1) · Cyclosporine (1) · Gemfibrozil (1) · Mycophenolic Acid (1) · Palliative Care (1)
Children's Hospital of Philadelphia (2) · Forge Biologics, Inc (2) · Masonic Cancer Center, University of Minnesota (2) · University of Pittsburgh (2) · CENTOGENE GmbH Rostock (1) · Polaryx Therapeutics, Inc. (1) · RTI International (1) · State University of New York at Buffalo (1) · University of Chicago (1)
United States (11) · Egypt (1) · Germany (1) · India (1) · Sri Lanka (1)
Adeline Vanderver, MD (2) · Paul Orchard, MD (2) · Chester B. Whitley, Ph.D., M.D. (1) · Curt Scharfe, MD, PhD (1) · Deepa S Rajan, MD (1) · Michael Msall, M.D. (1) · Nancy Lyon, CPNP (1) · Patricia K. Duffner, M.D. (1) · Peter Bauer, Prof. (1) · Thomas J. Langan, MD (1)
PMID 21586746, PMID 31315600, PMID 32342562, PMID 32749716, PMID 35142618, PMID 36196048, PMID 39499628
R01HD104814 (1), R01NS061965 (1), U01NS106845 (1), U54NS065768 (1), U54NS115052 (1)